CY1124323T1 - Βελτιωμενες μεθοδοι για τον καθαρισμο ανασυνδυασμενων φορεων aav - Google Patents

Βελτιωμενες μεθοδοι για τον καθαρισμο ανασυνδυασμενων φορεων aav

Info

Publication number
CY1124323T1
CY1124323T1 CY20211100639T CY211100639T CY1124323T1 CY 1124323 T1 CY1124323 T1 CY 1124323T1 CY 20211100639 T CY20211100639 T CY 20211100639T CY 211100639 T CY211100639 T CY 211100639T CY 1124323 T1 CY1124323 T1 CY 1124323T1
Authority
CY
Cyprus
Prior art keywords
purification
aav vectors
recombinant aav
improved methods
recombinant
Prior art date
Application number
CY20211100639T
Other languages
English (en)
Inventor
Paulene Mclean Quigley Sheldon
Gina Nichols
Barbara A. Thorne
Peter S. Gagnon
Original Assignee
Genzyme Corporation
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Genzyme Corporation filed Critical Genzyme Corporation
Publication of CY1124323T1 publication Critical patent/CY1124323T1/el

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • C12N7/02Recovery or purification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • C12N15/861Adenoviral vectors
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N30/00Investigating or analysing materials by separation into components using adsorption, absorption or similar phenomena or using ion-exchange, e.g. chromatography or field flow fractionation
    • G01N30/02Column chromatography
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151Methods of production or purification of viral material
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N30/00Investigating or analysing materials by separation into components using adsorption, absorption or similar phenomena or using ion-exchange, e.g. chromatography or field flow fractionation
    • G01N30/02Column chromatography
    • G01N2030/022Column chromatography characterised by the kind of separation mechanism
    • G01N2030/027Liquid chromatography

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Immunology (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Analytical Chemistry (AREA)
  • Pathology (AREA)
  • General Physics & Mathematics (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Treatment Of Liquids With Adsorbents In General (AREA)
  • Medicinal Preparation (AREA)

Abstract

Στο παρόν παρέχονται μέθοδοι για τον καθαρισμό ανασυνδυασμένων αδενο-σχετιζόμενων ιικών φορέων (rAAV) που μπορεί να χρησιμοποιηθούν για γονιδιακή μεταφορά και συγκεκριμένα για γονιδιακή θεραπεία ή εμβολιασμό. Ανασυνδυασμένοι AAV φορείς της εφεύρεσης είναι ουσιαστικά ελεύθεροι από προσμίξεις κατά τη διεργασία, συμπεριλαμβανομένων συστατικών παραγωγής, όπως κυτταρικών νουκλεϊκών οξέων, κυτταρικών πρωτεϊνών, βοηθητικού ιού, και συστατικών του μέσου.
CY20211100639T 2009-06-16 2021-07-14 Βελτιωμενες μεθοδοι για τον καθαρισμο ανασυνδυασμενων φορεων aav CY1124323T1 (el)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US18760109P 2009-06-16 2009-06-16
EP16166278.8A EP3067417B1 (en) 2009-06-16 2010-06-16 Improved methods for purification of recombinant aav vectors
PCT/US2010/038897 WO2010148143A1 (en) 2009-06-16 2010-06-16 Improved methods for purification of recombinant aav vectors
EP10790155.5A EP2443233B1 (en) 2009-06-16 2010-06-16 Improved methods for purification of recombinant aav vectors

Publications (1)

Publication Number Publication Date
CY1124323T1 true CY1124323T1 (el) 2022-07-22

Family

ID=43356746

Family Applications (3)

Application Number Title Priority Date Filing Date
CY20161100843T CY1117936T1 (el) 2009-06-16 2016-08-29 Βελτιωμενες μεθοδοι για τον καθαρισμο ανασυνδυασμενων φορεων aav
CY20181101103T CY1121442T1 (el) 2009-06-16 2018-10-25 Βελτιωμενες μεθοδοι για τον καθαρισμο ανασυνδυασμενων φορεων aav
CY20211100639T CY1124323T1 (el) 2009-06-16 2021-07-14 Βελτιωμενες μεθοδοι για τον καθαρισμο ανασυνδυασμενων φορεων aav

Family Applications Before (2)

Application Number Title Priority Date Filing Date
CY20161100843T CY1117936T1 (el) 2009-06-16 2016-08-29 Βελτιωμενες μεθοδοι για τον καθαρισμο ανασυνδυασμενων φορεων aav
CY20181101103T CY1121442T1 (el) 2009-06-16 2018-10-25 Βελτιωμενες μεθοδοι για τον καθαρισμο ανασυνδυασμενων φορεων aav

Country Status (24)

Country Link
US (3) US10017746B2 (el)
EP (4) EP3067417B1 (el)
JP (3) JP5956331B2 (el)
KR (2) KR101812813B1 (el)
CN (2) CN102803478B (el)
BR (1) BRPI1015176B1 (el)
CA (2) CA3077531C (el)
CY (3) CY1117936T1 (el)
DK (2) DK3444335T3 (el)
ES (3) ES2693194T3 (el)
HK (1) HK1165830A1 (el)
HR (2) HRP20181743T1 (el)
HU (3) HUE028341T2 (el)
IL (3) IL216960A (el)
LT (2) LT3444335T (el)
MX (2) MX352986B (el)
PL (2) PL2443233T3 (el)
PT (3) PT3444335T (el)
RS (2) RS57784B1 (el)
RU (1) RU2016119739A (el)
SG (2) SG10201403290SA (el)
SI (2) SI3067417T1 (el)
TR (1) TR201815937T4 (el)
WO (1) WO2010148143A1 (el)

Families Citing this family (96)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9198984B2 (en) 2006-04-28 2015-12-01 The Trustees Of The University Of Pennsylvania Scalable production method for AAV
SI3067417T1 (sl) * 2009-06-16 2018-11-30 Genzyme Corporation Izboljšani postopki čiščenja vektorjev rekombinantnega AAV
EP2524215B1 (en) 2010-01-15 2018-12-26 Bio-Rad Laboratories, Inc. Surface neutralization of apatite
WO2014127314A1 (en) * 2013-02-15 2014-08-21 Phynexus, Inc. Devices and methods for purification of biological cells
US8895707B2 (en) 2010-08-18 2014-11-25 Bio-Rad Laboratories, Inc. Elution of proteins from hydroxyapatite resins without resin deterioration
US9592540B2 (en) 2011-02-02 2017-03-14 Bio-Rad Laboratories, Inc. Apatite surface neutralization with alkali solutions
JP5984854B2 (ja) 2011-03-16 2016-09-06 エフ.ホフマン−ラ ロシュ アーゲーF. Hoffmann−La Roche Aktiengesellschaft 移動相の調整によって、ポリペプチド単量体、凝集物およびフラグメントを分離するための選択性を向上させた、イオン交換クロマトグラフィー
US9610354B2 (en) * 2011-04-18 2017-04-04 National Center Of Neurology And Psychiatry Drug delivery particle and method for producing the same
BR112014005255A2 (pt) * 2011-09-08 2017-04-04 Uniqure Ip Bv remoção de vírus contaminante de preparações aav
CN104507952B (zh) 2012-05-30 2018-08-10 生物辐射实验室股份有限公司 基于磷灰石的色谱树脂的原位恢复
JP6200949B2 (ja) * 2012-05-31 2017-09-20 エイジェンシー フォー サイエンス,テクノロジー アンド リサーチ 固相ウレイドに対する生物学的標的物の選択的結合
EP2970920B1 (en) 2013-03-15 2018-04-25 The Children's Hospital of Philadelphia Scalable manufacturing process to produce recombinant lentiviral vectors in serum-free suspension cell culture system
US11332719B2 (en) 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
EP3741385A1 (en) 2013-04-17 2020-11-25 Genzyme Corporation Compositions for use in a method of treating and preventing macular degeneration
JP5861001B2 (ja) * 2013-07-11 2016-02-16 タカラバイオ株式会社 非エンベロープウイルスの製造方法
CN111068072A (zh) 2014-02-06 2020-04-28 建新公司 用于治疗和预防黄斑变性的组合物及方法
DK3137497T3 (da) 2014-05-02 2021-07-12 Genzyme Corp Aav-vektorer til retinal- og cns-genterapi
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
WO2015200282A1 (en) 2014-06-23 2015-12-30 Bio-Rad Laboratories, Inc. Apatite pretreatment
WO2015200299A2 (en) 2014-06-23 2015-12-30 Bio-Rad Laboratories, Inc. Apatite in-situ restoration
EP3168298B1 (en) 2014-07-10 2019-04-17 Takara Bio Inc. Production method for non-enveloped virus particles
CN104232687A (zh) * 2014-09-17 2014-12-24 许瑞安 一种重组腺相关病毒rAAV载体的分离纯化方法
BR112017009497A2 (pt) 2014-11-05 2018-02-06 Voyager Therapeutics, Inc. polinucleotídeos de aadc para o tratamento da doença de parkinson
KR20230145206A (ko) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
BR112017010087A2 (pt) 2014-11-14 2018-06-05 Voyager Therapeutics, Inc. composições e métodos para tratar esclerose lateral amiotrófica (ela)
EP3230441A4 (en) 2014-12-12 2018-10-03 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
KR102493350B1 (ko) 2015-01-09 2023-01-27 다카라 바이오 가부시키가이샤 비엔벨로프 바이러스 입자의 제조방법
EA201791630A1 (ru) 2015-01-20 2018-04-30 Джензим Корпорейшн Аналитическое ультрацентрифугирование для определения характеристик рекомбинантных вирусных частиц
EP3054007A1 (en) 2015-02-09 2016-08-10 Institut National De La Sante Et De La Recherche Medicale (Inserm) Recombinant adeno-associated virus particle purification comprising an immuno-affinity purification step
EP3054006A1 (en) * 2015-02-09 2016-08-10 Institut National De La Sante Et De La Recherche Medicale (Inserm) Recombinant adeno-associated virus particle purification with multiple-step anion exchange chromatography
SG11201706445SA (en) 2015-02-10 2017-09-28 Genzyme Corp Enhanced delivery of viral particles to the striatum and cortex
TWI707951B (zh) 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造
CN105907729B (zh) * 2015-05-05 2019-08-16 广东东阳光药业有限公司 一种制备人乳头瘤病毒l1蛋白类病毒样颗粒的方法
US20170067028A1 (en) * 2015-05-15 2017-03-09 Douglas J. Ballon Radiolabeling of adeno associated virus
US11896651B2 (en) 2015-05-16 2024-02-13 Genzyme Corporation Gene editing of deep intronic mutations
US9663766B2 (en) * 2015-07-24 2017-05-30 Bio-Rad Laboratories, Inc. Methods for purifying adenovirus vectors
US11015173B2 (en) 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV1
WO2017160360A2 (en) 2015-12-11 2017-09-21 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
EP3992283A1 (en) 2015-12-11 2022-05-04 The Trustees Of The University Of Pennsylvania Scalable purification method for aavrh10
EP3387117B1 (en) 2015-12-11 2022-11-23 The Trustees Of The University Of Pennsylvania Scalable purification method for aav8
BR112018012015A2 (pt) 2015-12-15 2018-12-04 Genzyme Corp vetores virais adeno-associados para tratar mucolipidose tipo ii
WO2017112948A1 (en) * 2015-12-24 2017-06-29 University Of Florida Research Foundation, Inc. Improved aav production using suspension adapted cells
ES2938833T3 (es) * 2016-03-28 2023-04-17 Ultragenyx Pharmaceutical Inc Métodos de inactivación térmica de adenovirus
PT3436051T (pt) * 2016-03-31 2021-11-02 Spark Therapeutics Inc Processo de fabrico de raav com base em coluna totalmente escalável
WO2017189959A1 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR THE TREATMENT OF DISEASES
GB201608046D0 (en) 2016-05-09 2016-06-22 Cambridge Entpr Ltd And Syndey Children S Hospitals Network Randwick And Westmead Incorporating The Treatment of complement-mediated disorders
MX2018014154A (es) 2016-05-18 2019-05-06 Voyager Therapeutics Inc Polinucleotidos moduladores.
RU2764587C2 (ru) 2016-05-18 2022-01-18 Вояджер Терапьютикс, Инк. Способы и композиции для лечения хореи гентингтона
GB201612248D0 (en) * 2016-07-14 2016-08-31 Puridify Ltd New process
TN2019000047A1 (en) 2016-08-15 2020-07-15 Genzyme Corp Methods for detecting aav
JP2019529350A (ja) 2016-08-16 2019-10-17 リジェネロン・ファーマシューティカルズ・インコーポレイテッドRegeneron Pharmaceuticals, Inc. 混合物から個々の抗体を定量する方法
JP2019530737A (ja) 2016-08-23 2019-10-24 アコーオス インコーポレイテッド ヒト対象において非加齢性聴力障害を治療するための組成物および方法
EP3831281A1 (en) 2016-08-30 2021-06-09 The Regents of The University of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
US11795473B2 (en) 2016-10-14 2023-10-24 Ultragenyx Pharmaceutical Inc. Use of tonicifying agents to enhance recombinant adeno-associated virus yield
HUE059631T2 (hu) 2016-10-25 2022-12-28 Regeneron Pharma Eljárások és összeállítások kromatográfiás adatelemzéshez
US11000561B2 (en) * 2016-11-04 2021-05-11 Takeda Pharmaceutical Company Limited Adeno-associated virus purification methods
US10626376B2 (en) * 2016-11-14 2020-04-21 St. Jude Children's Research Hospital Method for isolating and purifying adeno-associated virus particles using salt
WO2018175775A1 (en) 2017-03-22 2018-09-27 Dimension Therapeutics Cell culture methods involving hdac inhibitors or rep proteins
EP3619308A4 (en) 2017-05-05 2021-01-27 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT FOR HUNTINGTON'S MORBUS
AU2018261790A1 (en) 2017-05-05 2019-11-28 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
SG11201913157RA (en) * 2017-06-30 2020-01-30 Spark Therapeutics Inc Aav vector column purification methods
WO2019018342A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. NETWORK EQUIPMENT TRACK GUIDE SYSTEM
WO2019028306A2 (en) 2017-08-03 2019-02-07 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS FOR ADMINISTRATION OF ADENO-ASSOCIATED VIRUSES
EP4124658A3 (en) 2017-10-16 2023-04-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
CN111655285A (zh) 2017-12-29 2020-09-11 百深公司 腺相关病毒的纯化方法
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
WO2019161365A1 (en) 2018-02-19 2019-08-22 Homology Medicines, Inc. Adeno-associated virus compositions for restoring f8 gene function and methods of use thereof
EP3787771A1 (en) * 2018-04-29 2021-03-10 REGENXBIO Inc. Scalable clarification process for recombinant aav production
TW202005694A (zh) 2018-07-02 2020-02-01 美商里珍納龍藥品有限公司 自混合物製備多肽之系統及方法
JP2021530548A (ja) * 2018-07-24 2021-11-11 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics, Inc. 遺伝子治療製剤を生産するための系および方法
JP7070238B2 (ja) * 2018-08-22 2022-05-18 株式会社島津製作所 液体クロマトグラフ分析装置、移動相供給装置、液体クロマトグラフ分析方法および移動相供給方法
MX2021003188A (es) * 2018-09-21 2021-07-16 Nightstarx Ltd Composiciones y métodos para la manufactura de vectores de terapia génica.
SG11202103614RA (en) 2018-10-12 2021-05-28 Genzyme Corp Generation of improved human pah for treatment of severe pku by liver-directed gene replacement therapy
EP4257155A3 (en) 2018-11-16 2024-02-28 Encoded Therapeutics, Inc. Compositions and methods for treating wilson's disease
EP3959325A2 (en) 2019-04-23 2022-03-02 INSERM (Institut National de la Santé et de la Recherche Médicale) New adeno-associated virus (aav) variants and uses thereof for gene therapy
US10653731B1 (en) 2019-07-15 2020-05-19 Vigene Biosciences Inc. Recombinantly-modified adeno-associated virus (rAAV) having improved packaging efficiency
US10801042B1 (en) 2019-07-15 2020-10-13 Vigene Biosciences, Inc. Use of ion concentrations to increase the packaging efficiency of recombinant adeno-associated virus
US10557149B1 (en) 2019-07-15 2020-02-11 Vigene Biosciences, Inc. Recombinantly-modified adeno-associated virus helper vectors and their use to improve the packaging efficiency of recombinantly-modified adeno-associated virus
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
CN115989234A (zh) 2020-01-29 2023-04-18 建新公司 用于眼部基因疗法的经修饰的腺相关病毒衣壳蛋白及其使用方法
BR112022016596A2 (pt) 2020-02-21 2022-11-16 Akouos Inc Composições e métodos para o tratamento de debilitação auditiva não associada à idade em um indivíduo humano
EP3919613A1 (en) * 2020-06-05 2021-12-08 Bia Separations D.O.O. Enhanced purification of adeno-associated virus to more effectively remove contaminating dna
JP6898535B1 (ja) * 2020-06-16 2021-07-07 HOYA Technosurgical株式会社 アパタイトカラムを使用したウイルスの精製方法
CN111808828B (zh) * 2020-09-14 2020-11-17 和元生物技术(上海)股份有限公司 腺相关病毒的分离纯化方法
WO2022072657A1 (en) 2020-10-01 2022-04-07 Genzyme Corporation Human pah expression cassette for treatment of pku by liver-directed gene replacement therapy
JP2022138144A (ja) 2021-03-09 2022-09-22 Jcrファーマ株式会社 組換えaav9ビリオンの製造方法
JP2024511409A (ja) 2021-03-22 2024-03-13 ジェンザイム・コーポレーション 空および完全aavキャプシドのサイズ排除クロマトグラフィー解析
WO2023201272A1 (en) 2022-04-12 2023-10-19 Genzyme Corporation Use of irak4 modulators for gene therapy
TW202402329A (zh) 2022-04-12 2024-01-16 美商健臻公司 Irak4調節劑於基因療法之用途(二)
TW202405430A (zh) 2022-04-12 2024-02-01 美商健臻公司 對於基因療法藥劑的先天免疫原性之樹突細胞試驗
US20230346977A1 (en) 2022-04-13 2023-11-02 Universitat Autònoma De Barcelona Treatment of neuromuscular diseases via gene therapy that expresses klotho protein
TW202403049A (zh) 2022-05-16 2024-01-16 美商健臻公司 治療異染性白質失養症之方法
WO2023240062A1 (en) 2022-06-07 2023-12-14 Adverum Biotechnologies, Inc. Melanopsin variants for vision restoration

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU722196B2 (en) * 1995-08-30 2000-07-27 Genzyme Corporation Chromatographic purification of adenovirus and AAV
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
EP2325299A3 (en) 1997-09-05 2011-10-05 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of recombinant AAV vectors
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
US7419817B2 (en) 2002-05-17 2008-09-02 The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography
PT1841863E (pt) * 2005-01-14 2010-10-25 Bayer Healthcare Llc Método para a purificação de factor vii
ES2559421T3 (es) * 2007-03-14 2016-02-12 Takeda Vaccines, Inc. Purificación de partículas similares a virus
GB2467320A (en) 2009-01-28 2010-08-04 Surrey Nanosystems Ltd Two methods of forming carbon nano-materials using filtered acetylene gas
SI3067417T1 (sl) * 2009-06-16 2018-11-30 Genzyme Corporation Izboljšani postopki čiščenja vektorjev rekombinantnega AAV

Also Published As

Publication number Publication date
US11840710B2 (en) 2023-12-12
JP6788056B2 (ja) 2020-11-18
EP2443233A4 (en) 2013-05-29
WO2010148143A1 (en) 2010-12-23
EP3444335A1 (en) 2019-02-20
CA3077531C (en) 2022-09-20
CY1117936T1 (el) 2017-05-17
US20190010468A1 (en) 2019-01-10
HUE028341T2 (en) 2016-12-28
KR20120047213A (ko) 2012-05-11
MX2011013613A (es) 2012-01-19
JP2016195600A (ja) 2016-11-24
HRP20181743T1 (hr) 2019-02-22
US10696952B2 (en) 2020-06-30
PT2443233T (pt) 2016-08-17
CA3077531A1 (en) 2010-12-23
CA2764176A1 (en) 2010-12-23
HK1165830A1 (zh) 2012-10-12
SG176283A1 (en) 2012-01-30
ES2588990T3 (es) 2016-11-08
RS57784B1 (sr) 2018-12-31
TR201815937T4 (tr) 2018-11-21
KR20180000341A (ko) 2018-01-02
CN105861454B (zh) 2020-03-03
EP2443233B1 (en) 2016-06-01
PT3067417T (pt) 2018-11-13
JP6496684B2 (ja) 2019-04-03
JP2019083822A (ja) 2019-06-06
ES2881050T3 (es) 2021-11-26
DK3444335T3 (da) 2021-07-26
IL216960A (en) 2017-07-31
IL253205B (en) 2018-07-31
PT3444335T (pt) 2021-07-12
HRP20211120T1 (hr) 2021-10-15
LT3444335T (lt) 2021-07-26
CN102803478B (zh) 2016-04-27
EP3067417B1 (en) 2018-07-25
SI3067417T1 (sl) 2018-11-30
CN102803478A (zh) 2012-11-28
CN105861454A (zh) 2016-08-17
RU2012101475A (ru) 2013-07-27
SG10201403290SA (en) 2014-10-30
CA2764176C (en) 2020-06-02
US20210139860A1 (en) 2021-05-13
BRPI1015176B1 (pt) 2019-10-29
HUE054940T2 (hu) 2021-10-28
LT3067417T (lt) 2018-11-12
BRPI1015176A2 (pt) 2017-09-26
US10017746B2 (en) 2018-07-10
IL253205A0 (en) 2017-08-31
CY1121442T1 (el) 2020-05-29
EP3444335B1 (en) 2021-04-14
SI3444335T1 (sl) 2021-08-31
RU2016119739A (ru) 2018-11-05
MX352986B (es) 2017-12-15
US20150024467A1 (en) 2015-01-22
HUE040636T2 (hu) 2019-03-28
PL3067417T3 (pl) 2019-02-28
JP2012529917A (ja) 2012-11-29
EP3919614A1 (en) 2021-12-08
EP3067417A3 (en) 2016-11-02
IL216960A0 (en) 2012-02-29
IL260215A (en) 2018-07-31
DK3067417T3 (en) 2018-11-19
EP3067417A2 (en) 2016-09-14
RS62086B1 (sr) 2021-08-31
JP5956331B2 (ja) 2016-07-27
PL2443233T3 (pl) 2017-08-31
ES2693194T3 (es) 2018-12-10
EP2443233A1 (en) 2012-04-25
KR101812813B1 (ko) 2017-12-27

Similar Documents

Publication Publication Date Title
CY1124323T1 (el) Βελτιωμενες μεθοδοι για τον καθαρισμο ανασυνδυασμενων φορεων aav
CO2020004738A2 (es) Variantes de cápsides de virus adenoasociados y métodos de uso de estas
MX2022006652A (es) Proteinas de capside modificadas para un suministro mejorado de vectores de parvovirus.
EA201391109A1 (ru) Молекулы нуклеиновой кислоты, кодирующие новые антигены герпеса, вакцина, включающая их, и способы их использования
CY1121132T1 (el) ΠΛΗΡΩΣ-ΑΝΘΡΩΠΙΝΑ ΜΟΝΟΚΛΩΝΙΚΑ ΑΝΤΙΣΩΜΑΤΑ ANTI-vap-1
EA202191418A1 (ru) Рекомбинантные вирусные векторы и нуклеиновые кислоты для их получения
JP2015096070A5 (el)
MX357803B (es) Moléculas de ácido nucleico artificiales.
WO2010138263A3 (en) Novel aav 's and uses thereof
MX362981B (es) Moleculas artificiales de acido nucleico para la expresion mejorada de proteina o peptido.
CY1115502T1 (el) Παραγοντες διεγερσης αποπτωσης για τη θεραπεια καρκινου και ανοσων και αυτοανοσων ασθενειων
DE602006019629D1 (de) Von zellen stammende virale impfstoffe mit geringen mengen an rest-zell-dna
WO2009124312A3 (en) Consensus sequences of chikungunya viral proteins, nucleic acid molecules encoding the same, and compositions and methods for using the same
WO2012145509A3 (en) Adeno-associated-virus rep sequences, vectors, and viruses
PT1685243E (pt) Linhas celulares aviárias imortalizadas para a produção de vírus
WO2012142434A3 (en) Aav mediated ctla-4 gene transfer to treat sjogren's syndrome
MX2019003615A (es) Proteina central y proteina del antigeno de superficie del virus de la hepatitis b y vacunas que comprenden estas.
MX343830B (es) Molecula de acido nucleico que codifica la proteina nuclear del virus de la hepatitis b y vacuna que comprende la misma.
CY1124707T1 (el) Συνθεσεις και μεθοδοι για την επαγωγη ενισχυμενης ανοσοαποκρισης με χρηση φορεων ιων poxvirus (ευλογοϊων)
DK3740222T3 (da) Modificeret RAAV-capsidprotein til genterapi
CL2012002900A1 (es) Secuencias de acido nucleico procedente de un virus causante del sindrome de miocardiopatia (cms) en peces; vector; celula huesped; vacuna de adn; proteina recombinante codificada; vacuna recombinante; inmunoensayo para la deteccion de dicho virus; kit de diagnostico; y uso de las mismas.
MX2016013584A (es) Parvovirus porcino.
EA201691981A1 (ru) Улучшенные транспортные молекулы модулярного антигена и их применение
PH12017501100A1 (en) Recombinant swinepox virus and vaccines
WO2015123592A3 (en) Recombinant rna particles and methods of use